Patch-Based Therapy Promotes Myelin Repair in Multiple Sclerosis

After inflammatory attacks, remyelination is often incomplete, leaving persistent neurological deficits. Existing therapies largely target inflammation and relapse prevention rather than tissue repair. To advance repair-focused treatment, researchers at Johannes Gutenberg University Mainz have developed a transdermal theophylline patch intended to stimulate myelin regeneration.

At Johannes Gutenberg University Mainz (JGU), investigators identified histone deacetylase 2 (HDAC2) as a key regulator of myelin repair and repurposed low-dose theophylline to activate this enzyme. In mouse studies, low-dose theophylline increased HDAC2 activity and supported remyelination. The approach leverages a long-established respiratory drug at substantially lower doses for a new neurological indication.

To achieve stable, low systemic exposure, JGU’s Department of Biopharmaceutics and Pharmaceutical Technology developed a specialized transdermal patch that releases small amounts of theophylline over several days. This delivery strategy is designed to sustain the concentration window associated with remyelination. Earlier preclinical work received EUR 140,000 in support from the Mainz Science Foundation.

ForTra gGmbH for Research Transfer, a nonprofit subsidiary of the Else Kröner-Fresenius Foundation, has awarded EUR 1.1 million to advance the patch toward clinical use. The funding enables technology transfer to a German patch manufacturer and optimization for larger-scale production under Good Manufacturing Practice (GMP) standards. Meeting GMP requirements is essential before human testing.

A Phase I trial at the Mainz University Medical Center will enroll healthy volunteers to evaluate patch adhesion, tolerability, and the efficiency of transdermal drug delivery. Efficacy in promoting remyelination will not be assessed at this stage. A multicenter Phase II study in Germany is planned, pending additional funding, to determine whether the approach promotes remyelination in patients with MS.

"With this funding, we are taking a decisive step toward our goal of translating our research findings into a potential treatment for people with MS," said Claire Jacob, Professor of Cellular Neurobiology at JGU.

"Our long-term goal is to develop a therapy that not only slows the progression of the disease, but also helps the nervous system repair damage that has already occurred," said Prof. Jacob.

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Johannes Gutenberg University Mainz